List of drugs granted breakthrough therapy designation

This is a list of drugs which granted breakthrough therapy designation by years:

Drugs may be listed more than once, as breakthrough designation is awarded individually for each indication.

2019

Drug	Manufacturer	Indication
Psilocybin	Usona Institute	Major depressive disorder[1]

2018

Drug	Manufacturer	Indication
Psilocybin	COMPASS Pathways	Treatment resistant depression[2][3]

2017

[4]

Drug	Manufacturer	Indication
Nivolumab	Bristol-Myers Squibb	Urothelial carcinoma
Ribociclib	Novartis	HR-positive, HER2-negative breast cancer
Pembrolizumab	Merck	Hodgkin Lymphoma
Avelumab	EMD Serono	metastatic Merkel cell carcinoma
Niraparib	Tesaro	recurrent ovarian cancer responsive to platinum-based chemotherapy
Ocrelizumab	Genentech	multiple sclerosis
Dupilumab	Regeneron Pharmaceuticals	atopic dermatitis
Palbociclib	Pfizer	HR-positive, HER2-negative breast cancer
Valbenazine	Neurocrine Biosciences	tardive dyskinesia
Cerliponase alpha	BioMarin	tripeptidyl peptidase 1 (TPP1) deficiency
Midostaurin	Novartis	FLT3-positive acute myeloid leukemia
Brigatinib	Ariad Pharmaceuticals	ALK-positive NSCLC
Durvalumab	AstraZeneca	metastatic urothelial carcinoma
Kisqali-Femara Co-pack	Novartis	breast cancer
Ribaxamase	Synthetic Biologics	antibiotic resistance / c. diff
Midomafetamine (Ecstasy)	Multidisciplinary Association for Psychedelic Studies	Posttraumatic stress disorder[5][6]

2016

Drug	Manufacturer	Indication
Elbasvir/grazoprevir	Merck	Hepatitis C
Palbociclib	Pfizer	HR-positive, HER2-negative breast cancer
Crizotinib	PF Prism	ROS-1-positive NSCLC
Venetoclax	AbbVie	CLL with 17p deletion
Cabozantinib	Exelixis	renal cell carcinoma
Pimavanserin	Acadia Pharmaceuticals	Parkinson's disease-related psychosis
Lenvatinib mesylate	Eisai	renal cell carcinoma
Nivolumab	Bristol-Myers Squibb	Hodgkin lymphoma
Atezolizumab	Genentech	urothelial carcinoma
Lumacaftor/ivacaftor	Vertex Pharmaceuticals	cystic fibrosis with CFTR F508del
Canakinumab	Novartis	Familial mediterranean fever
Canakinumab	Novartis	Hyper-IgD syndrome
Canakinumab	Novartis	TNF receptor associated periodic syndrome
Atezolizumab	Genentech	metastatic NSCLC
Olaratumab	Eli Lilly	soft tissue sarcoma
Pembrolizumab	Merck	metastatic NSCLC
Nivolumab	Bristol-Myers Squibb	head and neck cancer
Daratumumab	Janssen	multiple myeloma
Oliceridine	Trevena	Analgesia and pain management
Rucaparib	Clovis Oncology	BRCA-mutated ovarian cancer
Silver diamine fluoride	Elevate Oral Care	Early childhood caries

2015

Drug	Manufacturer	Indication
Ibrutinib	Pharmacyclics	Waldenstrom’s macroglobulinemia
Palbociclib	Pfizer	ER-positive, HER2-negative breast cancer
Ranibizumab	Genentech	Diabetic retinopathy
ivacaftor	Vertex Pharmaceuticals	cystic fibrosis with a variety of CFTR mutations
Aflibercept	Regeneron Pharmaceuticals	diabetic retinopathy
Sirolimus	PF Prism	lymphangioleiomyomatosis
Lumacaftor/ivacaftor	Vertex Pharmaceuticals	cystic fibrosis with CFTR F508del mutation
Ombitasvir/paritaprevir/ritonavir	AbbVie	Hepatitis C
Uridine triacetate	Wellstat	hereditary orotic aciduria
Pembrolizumab	Merck	PDL-1 positive NSCLC
Nivolumab	Bristol-Myers Squibb	metastatic NSCLC
Idarucizumab	Boehringer Ingelheim	reversal of dabigatran
Asfotase alfa	Alexion	perinatal/infantile/childhood hypophosphatasia
Osimertinib	AstraZeneca	EGFR-positive NSCLC
Daratumumab	Janssen	multiple myeloma
Nivolumab	Bristol-Myers Squibb	advanced renal cell carcinoma
Elotuzumab	Bristol-Myers Squibb	multiple myeloma
Sebelipase alfa	Synageva	lysosomal acid lipase deficiency
Alectinib	Roche	ALK-mutated NSCLC
Pembrolizumab	Merck	metastatic melanoma
Ibalizumab	TaiMed Biologics	HIV-1 infection

2014

Drug	Manufacturer	Indication
Ivacaftor	Vertex Pharmaceuticals	Cystic fibrosis with CFTR mutation
Ofatumumab	GlaxoSmithKline	Chronic lymphocytic leukemia (CLL)
Ceritinib	Novartis	ALK-positive NSCLC
Idelalisib	Gilead	Relapsed CLL
Ibrutinib	Pharmacyclics	CLL with 17p deletion
Eltrombopag	GlaxoSmithKline	aplastic anemia
Pembrolizumab	Merck	metastatic melanoma
Ledipasvir/sofosbuvir	Gilead	Hepatitis C
Nintedanib	Boehringer Ingelheim	idiopathic pulmonary fibrosis
Pirfenidone	Genentech	idiopathic pulmonary fibrosis
Blinatumomab	Amgen	Acute lymphoblastic leukemia
Ombitasvir/paritaprevir/ritonavir	AbbVie	Hepatitis C
Nivolumab	Bristol-Myers Squibb	metastatic melanoma
Ivacaftor	Vertex Pharmaceuticals	cystic fibrosis with CFTR R117H mutation

2013

Drug	Manufacturer	Indication
Obinutuzumab	Genentech	Chronic lymphocytic leukemia
Ibrutinib	Pharmacyclics	Mantle cell lymphoma
Sofosbuvir	Gilead	Hepatitis C

References

1. "FDA Grants Psilocybin Second Breakthrough Therapy Designation". Medscape. Retrieved 5 January 2020.
2. "COMPASS Pathways Receives FDA Breakthrough Therapy Designation for Psilocybin Therapy for Treatment-resistant Depression". www.compasspathways.com. October 23, 2018. Retrieved April 2, 2019.
3. "FDA Puts 'Magic Mushroom' Ingredient on Fast Track for Depression Treatment". www.healthline.com. November 7, 2018. Retrieved April 2, 2019.
4. "Approval report" (PDF). www.fda.gov. Retrieved 2019-06-12.
5. "FDA Grants Breakthrough Therapy Designation for MDMA-Assisted Psychotherapy for PTSD". maps.org. August 26, 2017. Retrieved April 2, 2019.
6. "Ecstasy as a Remedy for PTSD? You Probably Have Some Questions". www.nytimes.com. May 1, 2018. Retrieved April 2, 2019.